Akebia
Therapeutics, Inc. (NASDAQ: AKBA), a biopharmaceutical company
focused on delivering innovative therapies to patients with kidney
disease through the biology of hypoxia-inducible factor (HIF), today
announced the publication of positive results from a Phase 2b study of
vadadustat, a once-daily oral HIF stabilizer, in development for the
treatment of anemia related to chronic kidney disease (CKD). The study
demonstrated that vadadustat increased and maintained hemoglobin levels
in patients throughout the 20-week study in a predictable and controlled
manner. The peer-reviewed paper, titled “Vadadustat, a novel oral HIF
stabilizer, provides effective anemia treatment in
non-dialysis-dependent chronic kidney disease,” was published online
by Kidney International, the official journal of the
International Society of Nephrology.

“Treatment of anemia related to CKD with the current standard of care,
erythropoiesis-stimulating agents, can lead to substantial hemoglobin
increases above the desired target range,” said Pablo Pergola, M.D.,
Ph.D., Renal Associates PA, and University of Texas Health Science
Center at San Antonio. “These results demonstrate that vadadustat raised
and maintained hemoglobin levels while minimizing hemoglobin excursions
and improving iron mobilization, and may offer patients an effective
alternative therapy for treating renal anemia.”

The multicenter, randomized, double-blind, placebo-controlled study
assessed the ability of oral, once-daily vadadustat to correct anemia in
patients with stages 3a-5 non-dialysis dependent chronic kidney disease.
Patients initiated treatment with either 450 mg of vadadustat or
matching placebo, administered once daily for 20 weeks. The initial 450
mg daily dose of vadadustat was adjusted in accordance with the
patient's hemoglobin (Hb) response using a dose titration algorithm
designed to minimize Hb excursions above 13.0 g/dL. Patients were
assigned to one of three study groups: recombinant erythropoietin
stimulating agents (rESAs) treatment naïve, rESAs previously treated, or
rESAs actively treated. Within each group, patients were randomized 2:1
to receive vadadustat or placebo and were stratified according to CKD
stage and the presence or absence of diabetes mellitus. The full
manuscript is available on the Kidney International website at: http://www.kidney-international.org/article/S0085-2538(16)30357-X/fulltext.

Findings from the study include:

Vadadustat increased and maintained Hb levels in patients throughout
the study when compared to placebo; by week two, mean Hb levels in the
vadadustat group had increased significantly compared to placebo,
plateaued by week six to week eight and were sustained throughout the
20 weeks of treatment, with limited Hb excursions and dose adjustments;

Vadadustat improved iron mobilization compared to the placebo group,
as measured by significant decreases in hepcidin and ferritin, as well
as a significant increase in total iron binding capacity; and

There was no difference between the vadadustat and placebo groups in
the mean change from baseline in vascular endothelial growth factor
(VEGF) levels at week 12 or end of treatment.

Vadadustat was generally well tolerated in the Phase 2b trial and
overall adverse events were balanced between the treatment and placebo
groups.

“The positive results from our Phase 2b study provided a strong
foundation for our vadadustat global Phase 3 program, which continues to
enroll dialysis-dependent and non-dialysis patients with anemia related
to chronic kidney disease,” said Brad Maroni, M.D., Chief Medical
Officer at Akebia. “We will continue to publish the results from the
more than 15 clinical studies of vadadustat, and look forward to
reporting key findings from our Phase 3 program following the completion
of those trials.”

About Vadadustat

Vadadustat is an oral hypoxia-inducible factor (HIF) stabilizer with
best-in-class potential for the treatment of anemia related to chronic
kidney disease. Vadadustat, currently in development, exploits the same
mechanism of action used by the body to naturally adapt to lower oxygen
availability associated with a moderate increase in altitude. At higher
altitudes, the body responds to lower oxygen availability with increased
production of HIF, which coordinates the interdependent processes of
iron mobilization and erythropoietin production to increase red blood
cell production and, ultimately, improve oxygen delivery.

About Anemia Related to CKD

Approximately 30 million people in the U.S. have chronic kidney disease
(CKD), with an estimated 1.8 million of these patients suffering from
anemia. Anemia results from the body's inability to coordinate red blood
cell production in response to lower oxygen levels due to the
progressive loss of kidney function, which occurs in patients with CKD.
Left untreated, anemia significantly accelerates patients’ overall
deterioration of health with increased morbidity and mortality. Renal
anemia is currently treated with injectable recombinant erythropoiesis
stimulating agents, which are associated with inconsistent hemoglobin
responses and well-documented safety risks.

About Akebia Therapeutics

Akebia Therapeutics, Inc. is a biopharmaceutical company headquartered
in Cambridge, Massachusetts, focused on delivering innovative therapies
to patients with kidney disease through hypoxia-inducible factor
biology. Akebia’s lead product candidate, vadadustat, is an oral therapy
in development for the treatment of anemia related to chronic kidney
disease in both non-dialysis and dialysis patients. Akebia has commenced
its vadadustat Phase 3 Program, which includes the PRO2TECT
studies for non-dialysis patients with anemia secondary to chronic
kidney disease and INNO2VATE studies for dialysis-dependent
patients. For more information, please visit our website at www.akebia.com.

Forward-Looking Statements

This press release includes forward-looking statements. Such
forward-looking statements include those about Akebia's strategy, future
plans and prospects, including statements regarding the potential
indications and benefits of vadadustat, clinical development plans and
publication plans. The words “anticipate,” “appear,” “believe,”
“estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,”
“target,” “potential,” “will,” “would,” “could,” “should,” “continue,”
and similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain these
identifying words. Each forward-looking statement is subject to risks
and uncertainties that could cause actual results to differ materially
from those expressed or implied in such statement, including the risk
that existing preclinical and clinical data may not be predictive of the
results of ongoing or later clinical trials; the ability of Akebia to
successfully complete the clinical development of vadadustat; the
funding required to develop Akebia's product candidates and operate the
company, and the actual expenses associated therewith; the cost of the
Phase 3 studies of vadadustat and the availability of financing to cover
such costs; the timing and content of decisions made by the FDA and
other regulatory authorities; the rate of enrollment in clinical studies
of vadadustat; the actual time it takes to initiate and complete
clinical studies; the success of competitors in developing product
candidates for diseases for which Akebia is currently developing its
product candidates; and Akebia's ability to obtain, maintain and enforce
patent and other intellectual property protection for vadadustat. Other
risks and uncertainties include those identified under the heading “Risk
Factors” in Akebia's Annual Report on Form 10-Q for the quarter ended
June 30, 2016, and other filings that Akebia may make with the
Securities and Exchange Commission in the future. Akebia does not
undertake, and specifically disclaims, any obligation to update any
forward-looking statements contained in this press release.

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